CRISPR Treatment Prevents Hearing Loss in Mice
Researchers have successfully disrupted a gene variant that leads to deafness in mice, thus being able to restore their sense of hearing.
The treatment involved injection of a genome editing cocktail, which prevented progressive hearing loss in young animals, was conducted on mice at the Howard Hughes Medical Institute (HHMI), by Investigator David Liu and colleagues, according to a report published in journal Nature on December 20, 2017.
This is the first study that involved application of a genome editing approach to deafness in animals, Liu says., as this the study was performed in mice, implications for treating humans are still unclear. However, they hope that this work would someday help in the development of genetic deafness in humans, as well.
It has been found that around half of all deafness is a result of genetics, with limited treatment options. Until this research there was no technology available to directly treat the genetic mutation that sabotages hearing.
The gene—Tmcl, responsible for hearing, when undergoes even a single spelling error in its mutations, causes loss of hair cells in the inner ear, over time. This helps detect sound.
Scientists discovered that just one copy of mutation in Tmcl can cause hearing loss, however, destruction of mutated copy of a gene called Beethoven in mice, might preserve some hearing.
Researchers used the genome editing technology known as CRISPR-Cas9. The enzyme—Cas9, acts as molecular scissors, cutting through DNA double helix, which can ultimately disable a gene. They used an RNA guide to lead Cas9 to its target, however, over time the enzyme has the potential to cut other DNA that looks similar.
The team packaged Cas9 and the guide RNA into a greasy bundle that slips inside cells, they then made sure they do not stick around.
They designed a tool called base editor, which enables precision editing capabilities, disrupting, mutations linked to hearing loss disorders. They then placed the device on the head of mice, to monitor brain activity of regions involved in hearing. After four weeks, treated ears could hear sounds about 15 decibels lower than untreated ears.
This would prove to be the basis of further research in humans, as it has the potential to restore the capacity to hear.
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